First And Only FDA-Approved Type Ii Raf
Inhibitor For Patients With Relapsed Or Refractory Plgg Harboring A
Braf Fusion Or Rearrangement, Or Braf V600 Mutation Made
Possible Through PNOC Clinical Trial
FOLLOWING SUCCESSFUL FIREFLY-1 TRIAL IN
PARTNERSHIP WITH THE PNOC SCIENTIFIC CONSORTIUM AND DAY ONE
BIOPHARMACEUTICALS
SAN
RAFAEL, Calif., April 24,
2024 /PRNewswire/ -- The Pediatric
Neuro-Oncology Consortium Foundation (PNOC Foundation), a platinum
rated non-profit dedicated to supporting advancements in childhood
brain cancer research and clinical trials, announces the FDA
approval of Tovorafenib (DAY101), a significant advancement in the
fight against pediatric low-grade glioma brain tumors. This
approval is a direct result of the pivotal Phase 2 FIREFLY-1
trial/PNOC026, which demonstrated meaningful responses and a
favorable safety profile, marking a promising development in
pediatric neuro-oncology. This trial built on the initial PNOC
phase 1 trial, PNOC014.
This approval marks a significant milestone as the first and
only FDA-approved medicine for children with BRAF fusions or
rearrangements in pediatric low-grade glioma. Before Tovorafenib,
there was no standard of care for these children.
FIREFLY-1/PNOC026 underscored the effectiveness of Tovorafenib
(DAY101). This breakthrough therapy offers a great alternative
therapy option for children with low grade gliomas.
Dr Sabine Mueller, PNOC's
scientific co-founder said "PNOC is proud of our role in this
collaborative achievement and remains committed to advancing
research, enhancing treatment options, and improving the lives of
children affected by brain tumors. We extend our heartfelt
gratitude to all the patients, families, researchers, and
healthcare professionals who contributed to the success of PNOC014
and FIREFLY-1/PNOC026 and the development of Tovorafenib
(DAY101)."
"This is a landmark development for pediatric brain cancer
research and treatment," said PNOC Foundation co-founder and
patient parent Bruce Campbell. "It
shows what's possible when doctors, scientists, researchers,
biopharma, and philanthropists truly collaborate and work together.
This is a victory for kids and their families fighting pediatric
low-grade gliomas, however, there is still a huge unmet need. More
courage to invest in and fund new therapies and trials is needed to
bring further breakthroughs to other types of pediatric brain
cancer, the deadliest of childhood cancers."
"Seeing the promise of PNOC realized with advancements like
Tovorafenib confirms the crucial role of our foundation," stated
Allyn Campbell, co-founder of the
PNOC Foundation. "This milestone, made possible by our community's
support and the generosity of our donors, empowers us to continue
our quest to revolutionize pediatric neuro-oncology and improve the
lives of children affected by brain tumors."
For more information contact: media@pnocfoundation.org
To View the DayOne Press Release, Click HERE.
To View the FDA Press Release, Click HERE.
To learn more about the PNOC Foundation please
visit www.pnocfoundation.org.
About the PNOC Foundation:
The PNOC Foundation is a non-profit dedicated to supporting the
advancements in childhood brain cancer research. The PNOC
Foundation's vision is to lose no child to brain cancer, and to
improve the quality of life for those fighting and surviving
pediatric brain cancer. The PNOC Foundation was founded to support
the work of Pacific Pediatric Neuro-Oncology Consortium (PNOC),
formed in 2012 at UCSF, to provide access to personalized treatment
strategies and ultimately improve overall outcomes for children
with brain cancer and tumors.
Since its inception, PNOC has pushed beyond older paradigms and
historic trial designs, expanding nationally and globally to
accelerate clinical trial completion and find cures faster. Through
the cooperative open-source network, PNOC is fostering worldwide
collaboration with the world's leading pediatric brain tumor
specialists. In the pursuit of enhanced clinical trial development
and treatments for CNS cancer. PNOC has grown from a 7-member group
along the West Coast to a global consortium with 43 participating
sites, funded largely by philanthropy.
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content:https://www.prnewswire.com/news-releases/pnoc-foundation-a-leading-non-profit-to-further-advancements-in-pediatric-brain-cancer-announces-fda-approval-of-tovorafenib-day101-in-partnership-with-pnoc-scientific-consortium-and-day-one-biopharmaceuticals-302126588.html
SOURCE PNOC. Foundation